EA202190581A1 - Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a - Google Patents

Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a

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Publication number
EA202190581A1
EA202190581A1 EA202190581A EA202190581A EA202190581A1 EA 202190581 A1 EA202190581 A1 EA 202190581A1 EA 202190581 A EA202190581 A EA 202190581A EA 202190581 A EA202190581 A EA 202190581A EA 202190581 A1 EA202190581 A1 EA 202190581A1
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EA
Eurasian Patent Office
Prior art keywords
scn2a
scn1a
encephalopathy
treatment
sense oligonucleotides
Prior art date
Application number
EA202190581A
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English (en)
Inventor
Стивен Петру
Original Assignee
Рогкон, Инк.
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Publication date
Application filed by Рогкон, Инк. filed Critical Рогкон, Инк.
Publication of EA202190581A1 publication Critical patent/EA202190581A1/ru

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Abstract

В данном документе предложены способы, соединения и композиции для снижения экспрессии SCN2A у субъекта. Такие способы, соединения и композиции применимы для лечения, предотвращения, отсрочки или облегчения связанного с SCN1А заболевания или расстройства (например, синдрома Драве) у нуждающегося в этом субъекта.
EA202190581A 2018-08-20 2019-08-20 Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a EA202190581A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862765344P 2018-08-20 2018-08-20
PCT/US2019/047313 WO2020041348A1 (en) 2018-08-20 2019-08-20 Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies

Publications (1)

Publication Number Publication Date
EA202190581A1 true EA202190581A1 (ru) 2021-07-13

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EA202190581A EA202190581A1 (ru) 2018-08-20 2019-08-20 Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a

Country Status (9)

Country Link
US (1) US11939582B2 (ru)
EP (1) EP3840733A4 (ru)
JP (1) JP7476199B2 (ru)
CN (1) CN113329739A (ru)
AU (1) AU2019325255A1 (ru)
BR (1) BR112021003224A2 (ru)
CA (1) CA3110211A1 (ru)
EA (1) EA202190581A1 (ru)
WO (1) WO2020041348A1 (ru)

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US20230043648A1 (en) * 2020-02-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
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WO2020041348A1 (en) 2020-02-27
EP3840733A1 (en) 2021-06-30
BR112021003224A2 (pt) 2021-07-20
AU2019325255A1 (en) 2021-04-15
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